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Last Updated: December 31, 2025

CLINICAL TRIALS PROFILE FOR PEVONEDISTAT


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Clinical Trials for Pevonedistat

Trial ID Title Status Sponsor Phase Summary
NCT01814826 ↗ Study of MLN4924 Plus Azacitidine in Treatment-naive Participants With Acute Myelogenous Leukemia (AML) Who Are 60 Years or Older Completed Millennium Pharmaceuticals, Inc. Phase 1 The purpose of this study is to establish the maximum tolerated dose (MTD), and to assess the safety and tolerability of MLN4924 (pevonedistat) in combination with azacitidine in treatment naive participants with AML who were 60 years of age or older.
NCT01862328 ↗ Dose Escalation, Multi-arm Study of MLN4924 Plus Docetaxel, Gemcitabine, or Combination of Carboplatin and Paclitaxel in Participants With Solid Tumors Completed Millennium Pharmaceuticals, Inc. Phase 1 The purpose of this study is to establish the maximum tolerated dose (MTD) and assess the safety and tolerability of MLN4924 (pevonedistat) in combination with docetaxel, paclitaxel and carboplatin, and gemcitabine in participants with solid tumors.
NCT02610777 ↗ An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML) Completed Millennium Pharmaceuticals, Inc. Phase 2 The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with HR-MDS or CMML, or low-blast AML.
NCT02782468 ↗ A Study of Pevonedistat in Adult East Asian Participants Active, not recruiting Millennium Pharmaceuticals, Inc. Phase 1 The purpose of this study is to evaluate the safety and tolerability of pevonedistat administered as a single agent and in combination with azacitidine in adult east Asian participants with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).
NCT03009240 ↗ Pevonedistat and Decitabine in Treating Patients With High Risk Acute Myeloid Leukemia Active, not recruiting National Cancer Institute (NCI) Phase 1 This phase I trial studies the side effects and best dose of pevonedistat when given together with decitabine in treating patients with high risk acute myeloid leukemia. Pevonedistat and decitabine may stop the growth of cancer cells by blocking some of the enzymes need for cell growth.
NCT03009240 ↗ Pevonedistat and Decitabine in Treating Patients With High Risk Acute Myeloid Leukemia Active, not recruiting City of Hope Medical Center Phase 1 This phase I trial studies the side effects and best dose of pevonedistat when given together with decitabine in treating patients with high risk acute myeloid leukemia. Pevonedistat and decitabine may stop the growth of cancer cells by blocking some of the enzymes need for cell growth.
NCT03013998 ↗ Study of Biomarker-Based Treatment of Acute Myeloid Leukemia Recruiting Beat AML, LLC Phase 1/Phase 2 This screening and multi-sub-study Phase 1b/2 trial will establish a method for genomic screening followed by assigning and accruing simultaneously to a multi-study "Master Protocol (BAML-16-001-M1)." The specific subtype of acute myeloid leukemia will determine which sub-study, within this protocol, a participant will be assigned to evaluate investigational therapies or combinations with the ultimate goal of advancing new targeted therapies for approval. The study also includes a marker negative sub-study which will include all screened patients not eligible for any of the biomarker-driven sub-studies.
>Trial ID >Title >Status >Phase >Summary

Clinical Trial Conditions for Pevonedistat

Condition Name

Condition Name
Intervention Trials
Myelodysplastic Syndromes 5
Leukemia, Myeloid, Acute 5
Refractory Acute Myeloid Leukemia 4
Recurrent Acute Myeloid Leukemia 4
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Condition MeSH

Condition MeSH
Intervention Trials
Leukemia 19
Leukemia, Myeloid, Acute 17
Leukemia, Myeloid 16
Myelodysplastic Syndromes 12
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Clinical Trial Locations for Pevonedistat

Trials by Country

Trials by Country
Location Trials
United States 206
Spain 24
Japan 14
Italy 11
Poland 9
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Trials by US State

Trials by US State
Location Trials
Florida 13
California 13
New York 12
Texas 12
Ohio 11
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Clinical Trial Progress for Pevonedistat

Clinical Trial Phase

Clinical Trial Phase
Clinical Trial Phase Trials
Phase 3 2
Phase 2 10
Phase 1/Phase 2 5
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Clinical Trial Status

Clinical Trial Status
Clinical Trial Phase Trials
Active, not recruiting 13
Recruiting 9
Completed 6
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Clinical Trial Sponsors for Pevonedistat

Sponsor Name

Sponsor Name
Sponsor Trials
National Cancer Institute (NCI) 12
Millennium Pharmaceuticals, Inc. 12
Takeda 7
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Sponsor Type

Sponsor Type
Sponsor Trials
Other 24
Industry 20
NIH 12
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